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Fabio Candotti

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218 publications

Sous presse | 2024 | 2023 | 2022 | 2021 | 2020 | 2019 | 2018 | 2017 | 2016 | 2015 | 2014 | 2013 | 2012 | 2011 | 2010 | 2009 | 2008 | 2007 | 2006 | 2005 | 2004 | 2003 | 2002 | 2001 | 2000 | 1999 | 1998 | 1997 | 1996 | ...

Sous presse

Primary cutaneous T-cell lymphoma not otherwise specified (NOS) uncovering a novel RAB27A variant in Griscelli syndrome type 2.

Blanchard G., Atallah I., Blanchard M., Fehrenbacher B., Schaller M., Riccio O., Ballerini C., Candotti F., Guenova E. The British journal of dermatology. Peer-reviewed.

[DOI][Pmid][serval:BIB_FCE7997FD1E1]

2024

Safety and immunogenicity of CD40.HIVRI.Env, a dendritic cell-based HIV vaccine, in healthy HIV-uninfected adults: a first-in-human randomized, placebo-controlled, dose-escalation study (ANRS VRI06).

Levy Y., Moog C., Wiedemann A., Launay O., Candotti F., Hardel L., Durand M., Rieux V., Diallo A., Lacabaratz C. et al., 2024/11. EClinicalMedicine, 77 p. 102845. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_40E7F12D27D8]

Variants in IGLL1 cause a broad phenotype from agammaglobulinemia to transient hypogammaglobulinemia.

Soomann M., Bily V., Elgizouli M., Kraemer D., Akgül G., von Bernuth H., Bloomfield M., Brodszki N., Candotti F., Förster-Waldl E. et al., 2024/11. The Journal of allergy and clinical immunology, 154 (5) pp. 1313-1324.e7. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_FC5AAA31445F]

Immunoglobulin class-switch recombination: Mechanism, regulation, and related diseases.

Liu J.C., Zhang K., Zhang X., Guan F., Zeng H., Kubo M., Lee P., Candotti F., James L.K., Camara NOS et al., 2024/08. MedComm, 5 (8) pp. e662. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_BDDD0EE7D2F4]

The HyperPed-COVID international registry: Impact of age of onset, disease presentation and geographical distribution on the final outcome of MIS-C.

Caorsi R., Consolaro A., Speziani C., Sozeri B., Ulu K., Faugier-Fuentes E., Menchaca-Aguayo H., Ozen S., Sener S., Akhter Rahman S. et al., 2024/07. Journal of autoimmunity, 147 p. 103265. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_25A4B1A13301]

Clinical efficacy of SARS-CoV-2 Omicron-neutralizing antibodies in immunoglobulin preparations for the treatment of agammaglobulinemia in patients with primary antibody deficiency.

Karbiener M., Kindle G., Meyts I., Seppänen MRJ, Candotti F., Kamieniak M., Ilk R., Kreil T.R., Seidel M.G., ESID-COVID Consortium, 2024/06. Journal of medical virology, 96 (6) pp. e29738. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_3EE478B889BC]

2023

The role of dendritic cells in COVID-19 infection.

Wang X., Guan F., Miller H., Byazrova M.G., Cndotti F., Benlagha K., Camara NOS, Lei J., Filatov A., Liu C., 2023/12. Emerging microbes & infections, 12 (1) p. 2195019. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_9405D2DA3A70]

Correction to: New Dominant‑Negative IL6ST Variants Expand the Immunological and Clinical Spectrum of GP130‑Dependent Hyper‑IgE Syndrome.

Arlabosse T., Materna M., Riccio O., Schnider C., Angelini F., Perreau M., Rochat I., Superti-Furga A., Campos-Xavier B., Héritier S. et al., 2023/10. Journal of clinical immunology, 43 (7) p. 1674. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_D920DAB3A125]

New Dominant-Negative IL6ST Variants Expand the Immunological and Clinical Spectrum of GP130-Dependent Hyper-IgE Syndrome.

[URN][DOI][WoS][Pmid][serval:BIB_BB16C2AE927C]

Pediatric Allergy and Immunology (PAI) is for polishing with artificial intelligence, but careful use.

Eigenmann P., Akenroye A., Atanaskovic Markovic M., Candotti F., Ebisawa M., Genuneit J., Kalayci Ö., Kollmann D., Leung ASY, Peters R.L. et al., 2023/09. Pediatric allergy and immunology, 34 (9) pp. e14023. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_F053F1C1ECE1]

PSTPIP1-Associated Myeloid-Related Proteinemia Inflammatory (PAMI) Syndrome: A Systematic Review.

Mejbri M., Renella R., Candotti F., Jaques C., Holzinger D., Hofer M., Theodoropoulou K., 2023/08/19. Genes, 14 (8) p. 1655. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_E4AFB0AA68AA]

Gene Therapy for Inborn Errors of Immunity.

Arlabosse T., Booth C., Candotti F., 2023/06. The journal of allergy and clinical immunology. In practice, 11 (6) pp. 1592-1601. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_74E2CD2A10A2]

Severe combined immunodeficiency: improved survival leading to detection of underlying liver disease.

Vittal A., Abdul Majeed N., Garabedian E., Marko J., Kleiner D.E., Sokolic R., Candotti F., Malech H., Heller T., Koh C., 2023/05/19. BMC gastroenterology, 23 (1) p. 166. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_5F4596E72743]

Access to gene therapy for rare diseases when commercialization is not fit for purpose.

Fox T., Bueren J., Candotti F., Fischer A., Aiuti A., Lankester A., AGORA Initiative, Booth C., 2023/03. Nature medicine, 29 (3) pp. 518-519. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_E19E38D216AF]

"Inborn errors of immunity: An expanding horizon through a multitude of biological pathways".

Candotti F., Eigenmann P., 2023/01. Pediatric allergy and immunology, 34 (1) pp. e13908. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_1E194F423ED2]

2022

Correction to: Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients.

Hashem H., Bucciol G., Ozen S., Unal S., Bozkaya I.O., Akarsu N., Taskinen M., Koskenvuo M., Saarela J., Dimitrova D. et al., 2022/10..

[DOI][WoS][Pmid][serval:BIB_158647440B8D]

Editorial comment on "Inborn errors of immunity associated with defects of thymic development".

Beken B., Castagnoli R., Candotti F., 2022/09. Pediatric allergy and immunology, 33 (9) pp. e13847. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_EEF54F454139]

Common Variable Immunodeficiency in a Carrier of the ADA2 R169Q Variant: Coincidence or Causality?

Moi L., Schnider C., Riccio O., Hershfield M.S., Candotti F., 2022/07. Journal of clinical immunology, 42 (5) pp. 959-961. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_CB464CB7181B]

Déficit en adénosine désaminase 2 : une maladie aux présentations multiples [Adenosine deaminase 2 deficiency: a disease with multiple presentations]

Caratsch L., Schnider C., Moi L., Theodoropoulou K., Candotti F., Hofer M., 2022/04/06. Revue medicale suisse, 18 (776) pp. 669-673. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_D3726C7A7818]

Signaling networks in B cell development and related therapeutic strategies.

Ren A., Sun J., Yin W., Westerberg L.S., Miller H., Lee P., Candotti F., Guan F., Lei J., Gong Q. et al., 2022/04. Journal of leukocyte biology, 111 (4) pp. 877-891. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_B8683F218609]

Critical role of WASp in germinal center tolerance through regulation of B cell apoptosis and diversification.

Descatoire M., Fritzen R., Rotman S., Kuntzelman G., Leber X.C., Droz-Georget S., Thrasher A.J., Traggiai E., Candotti F., 2022/03/08. Cell reports, 38 (10) p. 110474. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_114B7364DECA]

B cell-T cell interplay in immune regulation: A focus on follicular regulatory T and regulatory B cell functions.

Tan D., Yin W., Guan F., Zeng W., Lee P., Candotti F., James L.K., Saraiva Camara N.O., Haeryfar SMM, Chen Y. et al., 2022. Frontiers in cell and developmental biology, 10 p. 991840. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_C05812F2D567]

Case Report: Susceptibility to viral infections and secondary hemophagocytic lymphohistiocytosis responsive to intravenous immunoglobulin as primary manifestations of adenosine deaminase 2 deficiency.

Drago E., Garbarino F., Signa S., Grossi A., Schena F., Penco F., Santori E., Candotti F., Boztug K., Volpi S. et al., 2022. Frontiers in immunology, 13 p. 937108. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_E9D9D3B88DB9]

The role of B cells in COVID-19 infection and vaccination.

Chen S., Guan F., Candotti F., Benlagha K., Camara NOS, Herrada A.A., James L.K., Lei J., Miller H., Kubo M. et al., 2022. Frontiers in immunology, 13 p. 988536. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_29D06720D861]

2021

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.

Reinhardt B., Habib O., Shaw K.L., Garabedian E., Carbonaro-Sarracino D.A., Terrazas D., Fernandez B.C., De Oliveira S., Moore T.B., Ikeda A.K. et al., 2021/10/14. Blood, 138 (15) pp. 1304-1316. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_EFBE68E0CAAF]

Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients.

Hashem H., Bucciol G., Ozen S., Unal S., Bozkaya I.O., Akarsu N., Taskinen M., Koskenvuo M., Saarela J., Dimitrova D. et al., 2021/10. Journal of clinical immunology, 41 (7) pp. 1633-1647. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_F136CAD06DF2]

Immune deficiency, autoimmune disease and intellectual disability: A pleiotropic disorder caused by biallelic variants in the TPP2 gene.

Atallah I., Quinodoz M., Campos-Xavier B., Peter V.G., Fouriki A., Bonvin C., Bottani A., Kumps C., Angelini F., Bellutti Enders F. et al., 2021/06. Clinical genetics, 99 (6) pp. 780-788. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_ED69F497CA09]

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

Kohn D.B., Booth C., Shaw K.L., Xu-Bayford J., Garabedian E., Trevisan V., Carbonaro-Sarracino D.A., Soni K., Terrazas D., Snell K. et al., 2021/05/27. The New England journal of medicine, 384 (21) pp. 2002-2013. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_B0DDF70ABF8F]

COVID-19 pédiatrique - Pathophysiologie, réponses immunitaires, prédispositions génétiques et syndrome hyperinflammatoire [Pediatric COVID-19 - Pathophysiology, immune responses, genetic predispositions, hyperinflammatory syndrome]

Blanchard-Rohner G., Pachlopnik Schmid J., Candotti F., Hofer M., 2021/02/17. Revue medicale suisse, 17 (726) pp. 334-337. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_7C258D1A476B]

Pédiatrie - Dépistage des nouveau-nés en Suisse pour les déficiences sévères à lymphocytes T et B [Newborn screening for severe T and B lymphocyte deficiencies in Switzerland]

Fouriki A., Schnider C., Theodoropoulou K., Pachlopnik J., Hofer M., Candotti F., 2021/01/13. Revue medicale suisse, 17 (720-1) pp. 68-76. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_35F00EC79F4E]

Novel Discoveries in Immune Dysregulation in Inborn Errors of Immunity.

Ren A., Yin W., Miller H., Westerberg L.S., Candotti F., Park C.S., Lee P., Gong Q., Chen Y., Liu C., 2021. Frontiers in immunology, 12 p. 725587. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_5ABC78DF7B11]

Transcriptomic Signature Differences Between SARS-CoV-2 and Influenza Virus Infected Patients.

Bibert S., Guex N., Lourenco J., Brahier T., Papadimitriou-Olivgeris M., Damonti L., Manuel O., Liechti R., Götz L., Tschopp J. et al., 2021. Frontiers in immunology, 12 p. 666163. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_91CEC9119F7E]

2020

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry.

Kuo C.Y., Garabedian E., Puck J., Cowan M.J., Sullivan K.E., Buckley R.H., Cunningham-Rundles C., Marsh R., Candotti F., Kohn D.B., 2020/11. Journal of clinical immunology, 40 (8) pp. 1124-1131. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_D4F5DD043A76]

Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.

Bradford K.L., Liu S., Krajinovic M., Ansari M., Garabedian E., Tse J., Wang X., Shaw K.L., Gaspar H.B., Candotti F. et al., 2020/10. Biology of blood and marrow transplantation, 26 (10) pp. 1819-1827. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_4BA60F08CDFC]

Intra-uterine growth restriction induced by maternal low-protein diet causes long-term alterations of thymic structure and function in adult male rat offspring.

Armengaud J.B., Dennebouy Z., Labes D., Fumey C., Wilson A., Candotti F., Yzydorczyk C., Simeoni U., 2020/04/28. The British journal of nutrition, 123 (8) pp. 892-900. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_CAF70B17A491]

2019

A model for reticular dysgenesis shows impaired sensory organ development and hair cell regeneration linked to cellular stress.

Rissone A., Jimenez E., Bishop K., Carrington B., Slevin C., Wincovitch S.M., Sood R., Candotti F., Burgess S.M., 2019/12/20. Disease models & mechanisms, 12 (12). Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_AE7ADE4503CB]

Efficacy and Adverse Events During Janus Kinase Inhibitor Treatment of SAVI Syndrome.

Volpi S., Insalaco A., Caorsi R., Santori E., Messia V., Sacco O., Terheggen-Lagro S., Cardinale F., Scarselli A., Pastorino C. et al., 2019/07. Journal of clinical immunology, 39 (5) pp. 476-485. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_399FE048032A]

Gene therapy for Wiskott-Aldrich syndrome: here to stay.

Candotti F., 2019/05. The Lancet. Haematology, 6 (5) pp. e230-e231. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_21FD1FD5EF0A]

2018

Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome.

Candotti F., 2018/01. Journal of clinical immunology, 38 (1) pp. 13-27. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_00549880C4AB]

2017

Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency

Sokolic Robert, Candotti Fabio, 2017/05/22. Expert Opinion on Orphan Drugs, 5 (6) pp. 477-485. Peer-reviewed.

[DOI][serval:BIB_121BF6844E7B]

Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.

Shaw K.L., Garabedian E., Mishra S., Barman P., Davila A., Carbonaro D., Shupien S., Silvin C., Geiger S., Nowicki B. et al., 2017/05/01. The Journal of clinical investigation, 127 (5) pp. 1689-1699. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_116142C43273]

Hypogammaglobulinémie transitoire de l’enfant [Transient hypogammaglobulinemia of infancy]

Bellutti Enders F., Conti F., Candotti F., Angelini F., 2017/04/05. Revue medicale suisse, 13 (557) pp. 739-742. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_32D9F981D90B]

Séquençage à haut débit : outil de diagnostic des déficits immunitaires héréditaires [Next generation sequencing : a diagnostic tool for inherited immune defects]

Droz-Georget S., Riccio O., Royer-Bertrand B., Superti-Furga A., Candotti F., 2017/04/05. Revue medicale suisse, 13 (557) pp. 763-766. Peer-reviewed.

[Pmid][serval:BIB_5CC64A51219F]

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Cooper A. R., Lill G. R., Shaw K., Carbonaro-Sarracino D. A., Davila A., Sokolic R., Candotti F., Pellegrini M., Kohn D. B., 2017. Blood, 129 (19) pp. 2624-2635.

[DOI][Pmid][serval:BIB_A984D9BE9F25]

2016

The long terminal repeat negative control region is a critical element for insertional oncogenesis after gene transfer into hematopoietic progenitors with Moloney murine leukemia viral vectors.

Ikawa Y., Uchiyama T., Jagadeesh G.J., Candotti F., 2016/11. Gene therapy, 23 (11) pp. 815-818. Peer-reviewed.

[DOI][Pmid][serval:BIB_620CBE85F0D3]

Detection of Reactive Oxygen Species Using MitoSOX and CellROX in Zebrafish

Rissone Alberto, Candotti Fabio, 2016/10/05. Bio-Protocol, 6 (19) pp. NA. Peer-reviewed.

[DOI][serval:BIB_C1F022A936E1]

FOXP3+ Tregs require WASP to restrain Th2-mediated food allergy.

Lexmond W.S., Goettel J.A., Lyons J.J., Jacobse J., Deken M.M., Lawrence M.G., DiMaggio T.H., Kotlarz D., Garabedian E., Sackstein P. et al., 2016/10/03. The Journal of clinical investigation, 126 (10) pp. 4030-4044. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_63F9DB0CD983]

Type I interferonopathies in pediatric rheumatology.

Volpi S., Picco P., Caorsi R., Candotti F., Gattorno M., 2016/03/04. Pediatric rheumatology online journal, 14 (1) p. 35. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_F53B15A22E70]

Advances of gene therapy for primary immunodeficiencies.

Candotti F., 2016. F1000research, 5 p. 31. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_7FF778D09709]

Combined T Cell and B Cell Deficiency – SCID Forms: T−B+

Candotti Fabio, 2016. pp. 360-368 dans Encyclopedia of Immunobiology, Academic Press.

[serval:BIB_C922879F01CD]

N-WASP is required for B-cell-mediated autoimmunity in Wiskott-Aldrich syndrome.

Volpi S., Santori E., Abernethy K., Mizui M., Dahlberg C.I., Recher M., Capuder K., Csizmadia E., Ryan D., Mathew D. et al., 2016. Blood, 127 (2) pp. 216-220. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_3AB2A62512A7]

2015

Broad spectrum of autoantibodies in patients with Wiskott-Aldrich syndrome and X-linked thrombocytopenia

Crestani E., Volpi S., Candotti F., Giliani S., Notarangelo L. D., Chu J., Aldave Becerra J. C., Buchbinder D., Chou J., Geha R. S. et al., 2015/11. J Allergy Clin Immunol, 136 (5) pp. 1401-4 e1-3.

[DOI][Pmid][serval:BIB_D109D46F555B]

Broad spectrum of autoantibodies in patients with Wiskott-Aldrich syndrome and X-linked thrombocytopenia.

Crestani E., Volpi S., Candotti F., Giliani S., Notarangelo L.D., Chu J., Aldave Becerra J.C., Buchbinder D., Chou J., Geha R.S. et al., 2015/11. The Journal of allergy and clinical immunology, 136 (5) pp. 1401-4.e1-3. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_0BEC1BCD2DAC]

Age-Dependent Defects of Regulatory B Cells in Wiskott-Aldrich Syndrome Gene Knockout Mice.

Yokoyama T., Yoshizaki A., Simon K.L., Kirby M.R., Anderson S.M., Candotti F., 2015. Plos One, 10 (10) pp. e0139729. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_E2770578213B]

Altered BCR and TLR signals promote enhanced positive selection of autoreactive transitional B cells in Wiskott-Aldrich syndrome.

Kolhatkar N.S., Brahmandam A., Thouvenel C.D., Becker-Herman S., Jacobs H.M., Schwartz M.A., Allenspach E.J., Khim S., Panigrahi A.K., Luning Prak E.T. et al., 2015. Journal of Experimental Medicine, 212 (10) pp. 1663-1677. Peer-reviewed.

[DOI][Pmid][serval:BIB_0830B2716C0B]

Assessment of Immature Platelet Fraction in the Diagnosis of Wiskott-Aldrich Syndrome.

Sokolic R., Oden N., Candotti F., 2015. Frontiers in Pediatrics, 3 p. 49. Peer-reviewed.

[URN][DOI][Pmid][serval:BIB_E2680ABFEEAF]

Impulse oscillometry identifies peripheral airway dysfunction in children with adenosine deaminase deficiency.

Komarow H.D., Sokolic R., Hershfield M.S., Kohn D.B., Young M., Metcalfe D.D., Candotti F., 2015. Orphanet Journal of Rare Diseases, 10 (1) p. 159. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_9D71F79E18A7]

In vitro functional correction of Hermansky-Pudlak Syndrome type-1 by lentiviral-mediated gene transfer.

Ikawa Y., Hess R., Dorward H., Cullinane A.R., Huizing M., Gochuico B.R., Gahl W.A., Candotti F., 2015. Molecular Genetics and Metabolism, 114 (1) pp. 62-65. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_4EAD5A280AFA]

Individualized iterative phenotyping for genome-wide analysis of loss-of-function mutations.

Johnston J.J., Lewis K.L., Ng D., Singh L.N., Wynter J., Brewer C., Brooks B.P., Brownell I., Candotti F., Gonsalves S.G. et al., 2015. American Journal of Human Genetics, 96 (6) pp. 913-925. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_53271133DDC9]

Lentiviral-mediated gene therapy restores B cell tolerance in Wiskott-Aldrich syndrome patients.

Pala F., Morbach H., Castiello M.C., Schickel J.N., Scaramuzza S., Chamberlain N., Cassani B., Glauzy S., Romberg N., Candotti F. et al., 2015. Journal of Clinical Investigation, 125 (10) pp. 3941-3951. Peer-reviewed.

[DOI][Pmid][serval:BIB_B13635B49D35]

NBAS mutations cause a multisystem disorder involving bone, connective tissue, liver, immune system, and retina.

Segarra N.G., Ballhausen D., Crawford H., Perreau M., Campos-Xavier B., van Spaendonck-Zwarts K., Vermeer C., Russo M., Zambelli P.Y., Stevenson B. et al., 2015. American Journal of Medical Genetics. Part A, 167A (12) pp. 2902-2912. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_D06FCFC24F20]

Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.

Otsu M., Yamada M., Nakajima S., Kida M., Maeyama Y., Hatano N., Toita N., Takezaki S., Okura Y., Kobayashi R. et al., 2015. Journal of Clinical Immunology, 35 (4) pp. 384-398. Peer-reviewed.

[DOI][WoS][Pmid][serval:BIB_4CC409618D1B]

Reticular dysgenesis-associated AK2 protects hematopoietic stem and progenitor cell development from oxidative stress.

Rissone A., Weinacht K.G., la Marca G., Bishop K., Giocaliere E., Jagadeesh J., Felgentreff K., Dobbs K., Al-Herz W., Jones M. et al., 2015. Journal of Experimental Medicine, 212 (8) pp. 1185-1202. Peer-reviewed.

[URN][DOI][WoS][Pmid][serval:BIB_43128B927D92]

2014

Severe Combined Immunodeficiencies

Villa Anna, Moshous Despina, de Villartay J.P., Notarangelo Luigi D., Candotti Fabio, 2014/08/12. pp. 87-141 dans Sullivan Kathleen, Stiehm E. Richard (eds.) Stiehm's Immune Deficiencies, Academic Press.

[serval:BIB_7415F32BF57E]

Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases

Candotti F., 2014/04. Int J Hematol, 99 (4) pp. 383-92.

[DOI][Pmid][serval:BIB_884A92DF0904]

Molecular and phenotypic abnormalities of B lymphocytes in patients with Wiskott-Aldrich syndrome

Simon K. L., Anderson S. M., Garabedian E. K., Moratto D., Sokolic R. A., Candotti F., 2014/03. J Allergy Clin Immunol, 133 (3) pp. 896-9 e4.

[DOI][Pmid][serval:BIB_29FB15CDE436]

Primary Immune Deficiency Treatment Consortium (PIDTC) report

Griffith L. M., Cowan M. J., Notarangelo L. D., Kohn D. B., Puck J. M., Pai S. Y., Ballard B., Bauer S. C., Bleesing J. J., Boyle M. et al., 2014/02. J Allergy Clin Immunol, 133 (2) pp. 335-47.

[DOI][Pmid][serval:BIB_554AF113865A]

Actionable diagnosis of neuroleptospirosis by next-generation sequencing

Wilson M. R., Naccache S. N., Samayoa E., Biagtan M., Bashir H., Yu G., Salamat S. M., Somasekar S., Federman S., Miller S. et al., 2014. N Engl J Med, 370 (25) pp. 2408-17.

[DOI][Pmid][serval:BIB_60D422028531]

Activated STING in a vascular and pulmonary syndrome

Liu Y., Jesus A. A., Marrero B., Yang D., Ramsey S. E., Sanchez G. A. M., Tenbrock K., Wittkowski H., Jones O. Y., Kuehn H. S. et al., 2014. N Engl J Med, 371 (6) pp. 507-518.

[DOI][Pmid][serval:BIB_0F81943EEA19]

Early-onset stroke and vasculopathy associated with mutations in ADA2

Zhou Q., Yang D., Ombrello A. K., Zavialov A. V., Toro C., Zavialov A. V., Stone D. L., Chae J. J., Rosenzweig S. D., Bishop K. et al., 2014. N Engl J Med, 370 (10) pp. 911-20.

[DOI][Pmid][serval:BIB_733A47C9AE63]

Nuclear role of WASp in gene transcription is uncoupled from its ARP2/3-dependent cytoplasmic role in actin polymerization

Sadhukhan S., Sarkar K., Taylor M., Candotti F., Vyas Y. M., 2014. J Immunol, 193 (1) pp. 150-60.

[DOI][Pmid][serval:BIB_00C3B4B4FAFB]

2013

Elevated IgE and atopy in patients treated for early-onset ADA-SCID

Lawrence M. G., Barber J. S., Sokolic R. A., Garabedian E. K., Desai A. N., O'Brien M., Jones N., Bali P., Hershfield M. S., Stone K. D. et al., 2013/12. J Allergy Clin Immunol, 132 (6) pp. 1444-6.

[DOI][Pmid][serval:BIB_951DCB761341]

The altered landscape of the human skin microbiome in patients with primary immunodeficiencies

Oh J., Freeman A. F., Nisc Comparative Sequencing Program, Park M., Sokolic R., Candotti F., Holland S. M., Segre J. A., Kong H. H., 2013/12. Genome Res, 23 (12) pp. 2103-14.

[DOI][Pmid][serval:BIB_203C49074702]

Gene Therapy

Candotti Fabio, Fischer Alain, 2013/11. dans Ochs Hans D., Smith C.I. Edvard, Puck Jennifer M. (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

[DOI][serval:BIB_9C212954A2EF]

Immunodeficiency due to defects of purine metablism

Hirschhorn Rochelle, Grunebaum Elias, Roiofman Chaim, Candotti Fabio, 2013/11. dans Ochs Hans D., Smith C.I. Edvard, Puck Jennifer M. (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

[DOI][serval:BIB_D87DEC65966A]

Severe Combined Immunodeficiency and Combined Immunodeficiency Due To Cytokine Signaling Defects (IL2RG, JAK3, IL7R, IL2RA, JAK3 and STAT5b)

Puck Jennifer M., Candotti Fabio, Notarangelo Luigi D., Roifman C.M, 2013/11. dans Ochs Hans D., Smith CI. Edvard, Puck Jennifer M. (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

[DOI][serval:BIB_34A3260B5787]

Aberrant glycosylation of IgA in Wiskott-Aldrich syndrome and X-linked thrombocytopenia

Shimizu M., Kanegane H., Wada T., Motoyoshi Y., Morio T., Candotti F., Yachie A., 2013/02. J Allergy Clin Immunol, 131 (2) pp. 587-90 e1-3.

[DOI][Pmid][serval:BIB_974C7CAADABF]

A novel function of RNAs arising from the long terminal repeat of human endogenous retrovirus 9 in cell cycle arrest

Xu L., Elkahloun A. G., Candotti F., Grajkowski A., Beaucage S. L., Petricoin E. F., Calvert V., Juhl H., Mills F., Mason K. et al., 2013/01. J Virol, 87 (1) pp. 25-36.

[DOI][Pmid][serval:BIB_D59B85B6906D]

Efficient methods for targeted mutagenesis in zebrafish using zinc-finger nucleases: data from targeting of nine genes using CompoZr or CoDA ZFNs

Sood R., Carrington B., Bishop K., Jones M., Rissone A., Candotti F., Chandrasekharappa S. C., Liu P., 2013. PLoS One, 8 (2) pp. e57239.

[DOI][Pmid][serval:BIB_C947E3E7D3CB]

Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector

Horino S., Uchiyama T., So T., Nagashima H., Sun S. L., Sato M., Asao A., Haji Y., Sasahara Y., Candotti F. et al., 2013. PLoS One, 8 (8) pp. e71594.

[DOI][Pmid][serval:BIB_DE48288DB40E]

Peptide library-based evaluation of T-cell receptor breadth detects defects in global and regulatory activation in human immunologic diseases

Barber J. S., Yokomizo L. K., Sheikh V., Freeman A. F., Garabedian E., van Dijk E., Sokolic R., Candotti F., Weng N. P., Sereti I. et al., 2013. Proc Natl Acad Sci U S A, 110 (20) pp. 8164-9.

[DOI][Pmid][serval:BIB_BF6A2C8F6B08]

Platelets from WAS patients show an increased susceptibility to ex vivo phagocytosis

Prislovsky A., Zeng X., Sokolic R. A., Garabedian E. N., Anur P., Candotti F., Strom T. S., 2013. Platelets, 24 (4) pp. 288-96.

[DOI][Pmid][serval:BIB_A583544F702C]

2012

Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome

Uchiyama T., Adriani M., Jagadeesh G. J., Paine A., Candotti F., 2012/06. Mol Ther, 20 (6) pp. 1270-9.

[DOI][Pmid][serval:BIB_D72F3A7FB19B]

Multicentric dermatofibrosarcoma protuberans in patients with adenosine deaminase-deficient severe combined immune deficiency

Kesserwan C., Sokolic R., Cowen E. W., Garabedian E., Heselmeyer-Haddad K., Lee C. C., Pittaluga S., Ortiz C., Baird K., Lopez-Terrada D. et al., 2012/03. J Allergy Clin Immunol, 129 (3) pp. 762-769 e1.

[DOI][Pmid][serval:BIB_FF1E37949097]

Development of IgA nephropathy-like glomerulonephritis associated with Wiskott-Aldrich syndrome protein deficiency

Shimizu M., Nikolov N. P., Ueno K., Ohta K., Siegel R. M., Yachie A., Candotti F., 2012/02. Clin Immunol, 142 (2) pp. 160-6.

[DOI][Pmid][serval:BIB_855819419053]

B cell-intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice

Recher M., Burns S. O., de la Fuente M. A., Volpi S., Dahlberg C., Walter J. E., Moffitt K., Mathew D., Honke N., Lang P. A. et al., 2012. Blood, 119 (12) pp. 2819-28.

[DOI][Pmid][serval:BIB_118932EEEB25]

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Candotti F., Shaw K. L., Muul L., Carbonaro D., Sokolic R., Choi C., Schurman S. H., Garabedian E., Kesserwan C., Jagadeesh G. J. et al., 2012. Blood, 120 (18) pp. 3635-46.

[DOI][Pmid][serval:BIB_DD88F93A69A7]

2011

Somatic mosaicism caused by monoallelic reversion of a mutation in T cells of a patient with ADA-SCID and the effects of enzyme replacement therapy on the revertant phenotype

Moncada-Velez M., Velez-Ortega A., Orrego J., Santisteban I., Jagadeesh J., Olivares M., Olaya N., Hershfield M., Candotti F., Franco J., 2011/11. Scand J Immunol, 74 (5) pp. 471-81.

[DOI][Pmid][serval:BIB_AD8F58EE8EA0]

Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells

Adriani M., Jones K. A., Uchiyama T., Kirby M. R., Silvin C., Anderson S. M., Candotti F., 2011. Blood, 117 (24) pp. 6608-11.

[DOI][Pmid][serval:BIB_B08045F8F84C]

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency

Sokolic R., Maric I., Kesserwan C., Garabedian E., Hanson I. C., Dodds M., Buckley R., Issekutz A. C., Kamani N., Shaw K. et al., 2011. Blood, 118 (10) pp. 2688-94.

[DOI][Pmid][serval:BIB_87C05A86BC9C]

2010

Somatic mosaicism in the Wiskott-Aldrich syndrome: molecular and functional characterization of genotypic revertants

Davis B. R., Yan Q., Bui J. H., Felix K., Moratto D., Muul L. M., Prokopishyn N. L., Blaese R. M., Candotti F., 2010/04. Clin Immunol, 135 (1) pp. 72-83.

[DOI][Pmid][serval:BIB_CCDB35D30104]

Genetics. Mosaicism--switch or spectrum?

Davis B. R., Candotti F., 2010. Science, 330 (6000) pp. 46-7.

[DOI][Pmid][serval:BIB_188D97FBB91C]

Nuclear role of WASp in the pathogenesis of dysregulated TH1 immunity in human Wiskott-Aldrich syndrome

Taylor M. D., Sadhukhan S., Kottangada P., Ramgopal A., Sarkar K., D'Silva S., Selvakumar A., Candotti F., Vyas Y. M., 2010. Sci Transl Med, 2 (37) pp. 37ra44.

[DOI][Pmid][serval:BIB_49073892EB29]

Systemic autoimmunity and defective Fas ligand secretion in the absence of the Wiskott-Aldrich syndrome protein

Nikolov N. P., Shimizu M., Cleland S., Bailey D., Aoki J., Strom T., Schwartzberg P. L., Candotti F., Siegel R. M., 2010. Blood, 116 (5) pp. 740-7.

[DOI][Pmid][serval:BIB_4EC7D63AB835]

2009

Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management

Griffith L. M., Cowan M. J., Notarangelo L. D., Puck J. M., Buckley R. H., Candotti F., Conley M. E., Fleisher T. A., Gaspar H. B., Kohn D. B. et al., 2009/12. J Allergy Clin Immunol, 124 (6) pp. 1152-60 e12.

[DOI][Pmid][serval:BIB_19A9087F9D9C]

Self-inactivating retroviral vector-mediated gene transfer induces oncogene activation and immortalization of primary murine bone marrow cells

Bosticardo M., Ghosh A., Du Y., Jenkins N. A., Copeland N. G., Candotti F., 2009/11. Mol Ther, 17 (11) pp. 1910-8.

[DOI][Pmid][serval:BIB_55095207F479]

Gene therapy fulfilling its promise

Kohn D. B., Candotti F., 2009. N Engl J Med, 360 (5) pp. 518-21.

[DOI][Pmid][serval:BIB_F5A6622AD338]

How I treat ADA deficiency

Gaspar H. B., Aiuti A., Porta F., Candotti F., Hershfield M. S., Notarangelo L. D., 2009. Blood, 114 (17) pp. 3524-32.

[DOI][Pmid][serval:BIB_01140FAB3805]

Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness

Lagresle-Peyrou C., Six E. M., Picard C., Rieux-Laucat F., Michel V., Ditadi A., Demerens-de Chappedelaine C., Morillon E., Valensi F., Simon-Stoos K. L. et al., 2009/01. Nat Genet, 41 (1) pp. 106-11.

[DOI][Pmid][serval:BIB_08985CDE10C0]

Revertant somatic mosaicism in the Wiskott-Aldrich syndrome

Davis B. R., Candotti F., 2009. Immunol Res, 44 (1-3) pp. 127-31.

[DOI][Pmid][serval:BIB_FF2DEB66B781]

2008

Somatic mosaicism in primary immune deficiencies

Wada T., Candotti F., 2008/12. Curr Opin Allergy Clin Immunol, 8 (6) pp. 510-4.

[DOI][Pmid][serval:BIB_06D797D1BA17]

Measurement of proliferative responses of cultured lymphocytes

Muul L. M., Silvin C., James S. P., Candotti F., 2008/08. Curr Protoc Immunol, Chapter 7 pp. Unit 7 10 1-7 10 24.

[DOI][Pmid][serval:BIB_26CA9F59800A]

Recent advances in gene therapy for severe congenital immunodeficiency diseases

Sokolic R., Kesserwan C., Candotti F., 2008/07. Curr Opin Hematol, 15 (4) pp. 375-80.

[DOI][Pmid][serval:BIB_39E5BDCC18B8]

Gene therapy for adenosine deaminase deficiency in the USA

Condotti Fabio, Kohn Donald B., 2008. pp. 258-259 dans Blood Cells, Molecules, and Diseases. Peer-reviewed.

[serval:BIB_479BEA71B519]

Unprecedented diversity of genotypic revertants in lymphocytes of a patient with Wiskott-Aldrich syndrome

Davis B. R., Dicola M. J., Prokopishyn N. L., Rosenberg J. B., Moratto D., Muul L. M., Candotti F., Michael Blaese R., 2008. Blood, 111 (10) pp. 5064-7.

[DOI][Pmid][serval:BIB_AE4A6A3FB3EF]

2007

The phenomenon of spontaneous genetic reversions in the Wiskott-Aldrich syndrome: a report of the workshop of the ESID Genetics Working Party at the XIIth Meeting of the European Society for Immunodeficiencies (ESID). Budapest, Hungary October 4-7, 2006

Stewart D. M., Candotti F., Nelson D. L., 2007/11. J Clin Immunol, 27 (6) pp. 634-9.

[DOI][Pmid][serval:BIB_E7F8B4A0733C]

Immune responses to gene-modified T cells

Muul L. M., Candotti F., 2007/10. Curr Gene Ther, 7 (5) pp. 361-8.

[Pmid][serval:BIB_75E913A1B646]

Impaired in vitro regulatory T cell function associated with Wiskott-Aldrich syndrome

Adriani M., Aoki J., Horai R., Thornton A. M., Konno A., Kirby M., Anderson S. M., Siegel R. M., Candotti F., Schwartzberg P. L., 2007/07. Clin Immunol, 124 (1) pp. 41-8.

[DOI][Pmid][serval:BIB_63469190065F]

The expression of Wiskott-Aldrich syndrome protein (WASP) is dependent on WASP-interacting protein (WIP)

Konno A., Kirby M., Anderson S. A., Schwartzberg P. L., Candotti F., 2007/02. Int Immunol, 19 (2) pp. 185-92.

[DOI][Pmid][serval:BIB_3CE5C0041051]

Bovine apolipoprotein B-100 is a dominant immunogen in therapeutic cell populations cultured in fetal calf serum in mice and humans

Sakamoto N., Tsuji K., Muul L. M., Lawler A. M., Petricoin E. F., Candotti F., Metcalf J. A., Tavel J. A., Lane H. C., Urba W. J. et al., 2007. Blood, 110 (2) pp. 501-8.

[DOI][Pmid][serval:BIB_73E36CEAD13C]

Cartilage hair hypoplasia mutations that lead to RMRP promoter inefficiency or RNA transcript instability

Nakashima E., Tran J. R., Welting T. J., Pruijn G. J., Hirose Y., Nishimura G., Ohashi H., Schurman S. H., Cheng J., Candotti F. et al., 2007. Am J Med Genet A, 143A (22) pp. 2675-81.

[DOI][Pmid][serval:BIB_5E9417861EBF]

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

Engel B. C., Podsakoff G. M., Ireland J. L., Smogorzewska E. M., Carbonaro D. A., Wilson K., Shah A., Kapoor N., Sweeney M., Borchert M. et al., 2007. Blood, 109 (2) pp. 503-6.

[DOI][Pmid][serval:BIB_04B977317058]

Structure-function analysis of the WIP role in T cell receptor-stimulated NFAT activation: evidence that WIP-WASP dissociation is not required and that the WIP NH2 terminus is inhibitory

Dong X., Patino-Lopez G., Candotti F., Shaw S., 2007. J Biol Chem, 282 (41) pp. 30303-10.

[DOI][Pmid][serval:BIB_7505FC5E0F72]

2006

Autosomal recessive severe combined immunodeficiency due to defects in cytokine signaling pthways

Candotti Fabio, Notarangelo Luigi D., 2006/10/05. pp. 137-152 dans Ochs Hans D., Smith C.I. Edvard, Puck Jennifer M. (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

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Gene therapy

Candotti Fabio, Fischer Alain, 2006/10/05. pp. 688-705 dans Ochs Hans D., Smith C.I. Edvard, Puck Jennifer M. (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

[serval:BIB_1C753FBE5B82]

Immunodeficiency due to defects of purine metablism

Candotti Fabio, Hirschhorn Rochelle, 2006/10/05. pp. 169-196 dans Ochs Hans D., Smith C.I. Edvard, Puck Jennifer M. (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press,.

[serval:BIB_A1D65B9F91A0]

Immunodeficiencies: injecting some safety into SCID gene therapy?

Candotti F., Roifman C., Puck J. M., 2006/05. Gene Ther, 13 (9) pp. 741-3.

[Pmid][serval:BIB_CA3FF63CF89B]

Functional interaction of common gamma-chain and growth hormone receptor signaling apparatus

Adriani M., Garbi C., Amodio G., Russo I., Giovannini M., Amorosi S., Matrecano E., Cosentini E., Candotti F., Pignata C., 2006. J Immunol, 177 (10) pp. 6889-95.

[Pmid][serval:BIB_F14808A9A50D]

Gene therapy: X-SCID transgene leukaemogenicity

Thrasher A. J., Gaspar H. B., Baum C., Modlich U., Schambach A., Candotti F., Otsu M., Sorrentino B., Scobie L., Cameron E. et al., 2006. Nature, 443 (7109) pp. E5-6; discussion E6-7.

[DOI][Pmid][serval:BIB_5306D04B7EF3]

Lessons from the Wiskott-Aldrich syndrome

Puck J. M., Candotti F., 2006. N Engl J Med, 355 (17) pp. 1759-61.

[DOI][Pmid][serval:BIB_ACFAF150D510]

2005

Haematopoietic Stem Cell Gene Therapy

Thrasher Adrian J., Candotti Fabio, 2005/11/15. dans Encyclopedia in Genetics, Genomics, Proteomics and Bioinformatics, John Wiley & Sons.

[DOI][serval:BIB_2A14F0E5BECF]

When eligibility criteria clash with personal treatment choice: a dilemma of clinical research

Wilfond Benjamin, Candotti Fabio, 2005/04/14. pp. 310-322 dans Kodish Eric David (eds.) Ethics and Reseach with Children, Oxford University Press .

[serval:BIB_99C7B4311E5B]

Jak3, severe combined immunodeficiency, and a new class of immunosuppressive drugs

Pesu M., Candotti F., Husa M., Hofmann S. R., Notarangelo L. D., O'Shea J. J., 2005/02. Immunol Rev, 203 pp. 127-42.

[DOI][Pmid][serval:BIB_D70349D04E75]

Analysis of T-cell repertoire diversity in Wiskott-Aldrich syndrome

Wada T., Schurman S. H., Garabedian E. K., Yachie A., Candotti F., 2005. Blood, 106 (12) pp. 3895-7.

[DOI][Pmid][serval:BIB_49A48109191D]

CXCL12 signaling is independent of Jak2 and Jak3

Moriguchi M., Hissong B. D., Gadina M., Yamaoka K., Tiffany H. L., Murphy P. M., Candotti F., O'Shea J. J., 2005. J Biol Chem, 280 (17) pp. 17408-14.

[DOI][Pmid][serval:BIB_3B3AAF18A1C0]

2004

Jak3 and the pathogenesis of severe combined immunodeficiency

O'Shea J. J., Husa M., Li D., Hofmann S. R., Watford W., Roberts J. L., Buckley R. H., Changelian P., Candotti F., 2004/07. Mol Immunol, 41 (6-7) pp. 727-37.

[DOI][Pmid][serval:BIB_8D9302364336]

Retroviral-mediated gene transfer restores IL-12 and IL-23 signaling pathways in T cells from IL-12 receptor beta1-deficient patients

Bosticardo M., Witte I., Fieschi C., Novelli F., Casanova J. L., Candotti F., 2004/06. Mol Ther, 9 (6) pp. 895-901.

[DOI][Pmid][serval:BIB_7EEC287157B4]

A convenient method for positive selection of retroviral producing cells generating vectors devoid of selectable markers

Xu L., Tsuji K., Mostowski H., Otsu M., Candotti F., Rosenberg A. S., 2004. J Virol Methods, 118 (1) pp. 61-7.

[DOI][Pmid][serval:BIB_4BA17140EE42]

A novel form of complete IL-12/IL-23 receptor beta1 deficiency with cell surface-expressed nonfunctional receptors

Fieschi C., Bosticardo M., de Beaucoudrey L., Boisson-Dupuis S., Feinberg J., Santos O. F., Bustamante J., Levy J., Candotti F., Casanova J. L., 2004. Blood, 104 (7) pp. 2095-101.

[DOI][Pmid][serval:BIB_78BD5FBBC6CC]

Autosomal recessive SCID caused by JAK3 deficiency

Candotti Fabio, Notarangelo Luigi Dd., 2004. pp. 453-455 dans Stiehm E.R., Ochs H.D., Winklestein J.A. (eds.) Immunologic Disorders in Infants and Children, 5th Ed, W.B. Saunders.

[serval:BIB_913C7C331414]

Clinical gene therapy trials for ADA deficiency and other immunodeficiencies

Candotti Fabio, 2004. pp. 33-64 dans Bertolotti Roger, Ozawa Keiya, Hammond Kirk (eds.) Progress in Gene Therapy: Pioneering Stem Cell/Gene Therapy Trials: Vol 2, V.S.P. Intl Science.

[serval:BIB_B9983A5ED65B]

Differential contribution of Wiskott-Aldrich syndrome protein to selective advantage in T- and B-cell lineages

Konno A., Wada T., Schurman S. H., Garabedian E. K., Kirby M., Anderson S. M., Candotti F., 2004. Blood, 103 (2) pp. 676-8.

[DOI][Pmid][serval:BIB_9D2580D8653A]

Engraftment potential of human amnion and chorion cells derived from term placenta

Bailo M., Soncini M., Vertua E., Signoroni P. B., Sanzone S., Lombardi G., Arienti D., Calamani F., Zatti D., Paul P. et al., 2004. Transplantation, 78 (10) pp. 1439-48.

[Pmid][serval:BIB_33B5817021F9]

Multiple patients with revertant mosaicism in a single Wiskott-Aldrich syndrome family

Wada T., Schurman S. H., Jagadeesh G. J., Garabedian E. K., Nelson D. L., Candotti F., 2004. Blood, 104 (5) pp. 1270-2.

[DOI][Pmid][serval:BIB_0CCA5CBF2F3A]

2003

American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells

Kohn D. B., Sadelain M., Dunbar C., Bodine D., Kiem H. P., Candotti F., Tisdale J., Riviere I., Blau C. A., Richard R. E. et al., 2003/08. Mol Ther, 8 (2) pp. 180-7.

[Pmid][serval:BIB_487CBB7C414A]

Autoimmunity in Wiskott-Aldrich syndrome

Schurman S. H., Candotti F., 2003/07. Curr Opin Rheumatol, 15 (4) pp. 446-53.

[Pmid][serval:BIB_D46D86B0E405]

Primary immunodeficiencies and the rheumatologist

O'Shea J. J., Holland S., Candotti F., 2003/07. Curr Opin Rheumatol, 15 (4) pp. 413-6.

[Pmid][serval:BIB_6314506B9AEF]

High incidence of lymphomas in a subgroup of Wiskott-Aldrich syndrome patients

Shcherbina A., Candotti F., Rosen F. S., Remold-O'Donnell E., 2003/05. Br J Haematol, 121 (3) pp. 529-30.

[Pmid][serval:BIB_D3EA4046CF00]

Second-site mutation in the Wiskott-Aldrich syndrome (WAS) protein gene causes somatic mosaicism in two WAS siblings

Wada T., Konno A., Schurman S. H., Garabedian E. K., Anderson S. M., Kirby M., Nelson D. L., Candotti F., 2003/05. J Clin Invest, 111 (9) pp. 1389-97.

[DOI][Pmid][serval:BIB_08D6DF65D7D4]

Evidence that the mouse 3' kappa light chain enhancer confers position-independent transgene expression in T- and B-lineage cells

Xu L., Tsuji K., Mostowski H., Candotti F., Rosenberg A., 2003. Hum Gene Ther, 14 (18) pp. 1753-64.

[DOI][Pmid][serval:BIB_6A912066BB62]

In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in Jak3 knock-out animals

McCauslin C. S., Wine J., Cheng L., Klarmann K. D., Candotti F., Clausen P. A., Spence S. E., Keller J. R., 2003. Blood, 102 (3) pp. 843-8.

[DOI][Pmid][serval:BIB_32F86AE35E09]

Molecular basis of severe combined immunodeficiency: Lessons from cytokine signaling pathways

Visconti Roberta, Candotti Fabio, O'Shea John J., 2003. pp. 279-306 dans Finkel T., Gutkind S. (eds.) Signal Transduction and Human Disease, Wiley-Liss.

[serval:BIB_863A45BB365A]

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial

Muul L. M., Tuschong L. M., Soenen S. L., Jagadeesh G. J., Ramsey W. J., Long Z., Carter C. S., Garabedian E. K., Alleyne M., Brown M. et al., 2003. Blood, 101 (7) pp. 2563-9.

[DOI][Pmid][serval:BIB_4C28133A4ED8]

Severe combined immunodeficiency (SCID) with JAK3 deficiency

Candotti Fabio, Notarangelo Luigi D., 2003. dans Puck Jennifer M. (eds.) UpToDate, UpToDate.

[serval:BIB_CF82A6F641DD]

SLAM-associated protein deficiency causes imbalanced early signal transduction and blocks downstream activation in T cells from X-linked lymphoproliferative disease patients

Sanzone S., Zeyda M., Saemann M. D., Soncini M., Holter W., Fritsch G., Knapp W., Candotti F., Stulnig T. M., Parolini O., 2003. J Biol Chem, 278 (32) pp. 29593-9.

[DOI][Pmid][serval:BIB_186E95B733F7]

2002

Cytokines and their role in lymphoid development, differentiation and homeostasis

Hofmann S. R., Ettinger R., Zhou Y. J., Gadina M., Lipsky P., Siegel R., Candotti F., O'Shea J. J., 2002/12. Curr Opin Allergy Clin Immunol, 2 (6) pp. 495-506.

[DOI][Pmid][serval:BIB_CCE98E664A6D]

Pharmacokinetics and organ distribution of N-methanocarbathymidine, a novel thymidine analog, in mice bearing tumors transduced with the herpes simplex thymidine kinase gene

Noy R., Ben-Zvi Z., Elezra M., Candotti F., Ford Jr H., Morris J. C., Marquez V. E., Johns D. G., Agbaria R., 2002/11. Cancer Chemother Pharmacol, 50 (5) pp. 360-6.

[DOI][Pmid][serval:BIB_615288793EDB]

Antitumor activity and metabolic activation of N-methanocarbathymidine, a novel thymidine analogue with a pseudosugar rigidly fixed in the northern conformation, in murine colon cancer cells expressing herpes simplex thymidine kinase

Noy R., Ben-Zvi Z., Manor E., Candotti F., Morris J. C., Ford H. Jr., Marquez V. E., Johns D. G., Agbaria R., 2002/06. Mol Cancer Ther, 1 (8) pp. 585-93.

[Pmid][serval:BIB_5D1E542EA6C6]

Molecular aspects of primary immunodeficiencies: lessons from cytokine and other signaling pathways

Candotti F., Notarangelo L., Visconti R., O'Shea J., 2002/05. J Clin Invest, 109 (10) pp. 1261-9.

[DOI][Pmid][serval:BIB_E026C81D6583]

Flow cytometry analysis of adenosine deaminase (ADA) expression: a simple and reliable tool for the assessment of ADA-deficient patients before and after gene therapy

Otsu M., Hershfield M. S., Tuschong L. M., Muul L. M., Onodera M., Ariga T., Sakiyama Y., Candotti F., 2002. Hum Gene Ther, 13 (3) pp. 425-32.

[DOI][Pmid][serval:BIB_0C7A57C6B8F1]

Gene therapy in infants with severe combined immunodeficiency

Otsu M., Candotti F., 2002. BioDrugs, 16 (4) pp. 229-39.

[Pmid][serval:BIB_837E3BAE5B32]

Immune response to fetal calf serum by two adenosine deaminase-deficient patients after T cell gene therapy

Tuschong L., Soenen S. L., Blaese R. M., Candotti F., Muul L. M., 2002. Hum Gene Ther, 13 (13) pp. 1605-10.

[DOI][Pmid][serval:BIB_297C83734133]

Reconstitution of lymphoid development and function in ZAP-70-deficient mice following gene transfer into bone marrow cells

Otsu M., Steinberg M., Ferrand C., Merida P., Rebouissou C., Tiberghien P., Taylor N., Candotti F., Noraz N., 2002. Blood, 100 (4) pp. 1248-56.

[DOI][Pmid][serval:BIB_2729DC49CBC9]

Retrovirus-mediated WASP gene transfer corrects Wiskott-Aldrich syndrome T-cell dysfunction

Wada T., Jagadeesh G. J., Nelson D. L., Candotti F., 2002. Hum Gene Ther, 13 (9) pp. 1039-46.

[DOI][Pmid][serval:BIB_D97FDC6AC988]

2001

Gene therapy for primary immune deficiencies

Otsu M., Wada T., Candotti F., 2001/12. Curr Opin Allergy Clin Immunol, 1 (6) pp. 497-501.

[Pmid][serval:BIB_DD848FF37D21]

Unexpected and variable phenotypes in a family with JAK3 deficiency

Frucht D. M., Gadina M., Jagadeesh G. J., Aksentijevich I., Takada K., Bleesing J. J., Nelson J., Muul L. M., Perham G., Morgan G. et al., 2001/12. Genes Immun, 2 (8) pp. 422-32.

[DOI][Pmid][serval:BIB_33661FADA28D]

Unexpected effects of FERM domain mutations on catalytic activity of Jak3: structural implication for Janus kinases

Zhou Y. J., Chen M., Cusack N. A., Kimmel L. H., Magnuson K. S., Boyd J. G., Lin W., Roberts J. L., Lengi A., Buckley R. H. et al., 2001/11. Mol Cell, 8 (5) pp. 959-69. Peer-reviewed.

[Pmid][serval:BIB_5FC4BE935F17]

Gene therapy for immunodeficiency

Candotti F., 2001/09. Curr Allergy Asthma Rep, 1 (5) pp. 407-15.

[Pmid][serval:BIB_A5F958A3B7D2]

Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model

Aviles Mendoza G. J., Seidel N. E., Otsu M., Anderson S. M., Simon-Stoos K., Herrera A., Hoogstraten-Miller S., Malech H. L., Candotti F., Puck J. M. et al., 2001/04. Mol Ther, 3 (4) pp. 565-73.

[DOI][Pmid][serval:BIB_184D3D7CDC66]

Biosynthetic ganciclovir triphosphate: its isolation and characterization from ganciclovir-treated herpes simplex thymidine kinase-transduced murine cells

Agbaria R., Candotti F., Kelley J. A., Hao Z., Johns D. G., Cooney D. A., Blaese R. M., Ford H. Jr., 2001. Biochem Biophys Res Commun, 289 (2) pp. 525-30.

[DOI][Pmid][serval:BIB_452AEF753A55]

Expansion of Hepatic and Hematopoietic Stem Cells Utilizing Mouse Embryonic Liver Explants

Monga S. P. S., Tang Y., Candotti F., Rashid A., Wildner O., Mishra B., Iqbal S., Mishra L., 2001/01. Cell Transplant, 10 (1) pp. 81-89.

[DOI][Pmid][serval:BIB_FA990B6806B3]

Gene Therapy for Inherited Immunodeficiencies

Candotti Fabio, Blaese R. Michael, 2001. pp. 580-596 dans Burakoff T.B., Rosen F.S., Strom T.B. (eds.) Therapeutic Immunology, Blackwell Science.

[serval:BIB_B10EEA85A276]

Lack of dominant-negative effects of a truncated gamma(c) on retroviral-mediated gene correction of immunodeficient mice

Otsu M., Sugamura K., Candotti F., 2001. Blood, 97 (6) pp. 1618-24.

[Pmid][serval:BIB_8E569B682068]

Somatic mosaicism in Wiskott--Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism

Wada T., Schurman S. H., Otsu M., Garabedian E. K., Ochs H. D., Nelson D. L., Candotti F., 2001. Proc Natl Acad Sci U S A, 98 (15) pp. 8697-702.

[DOI][Pmid][serval:BIB_23B41BB67651]

2000

Of genes and phenotypes: the immunological and molecular spectrum of combined immune deficiency. Defects of the gamma(c)-JAK3 signaling pathway as a model

Notarangelo L. D., Giliani S., Mazza C., Mella P., Savoldi G., Rodriguez-Perez C., Mazzolari E., Fiorini M., Duse M., Plebani A. et al., 2000/12. Immunol Rev, 178 pp. 39-48.

[Pmid][serval:BIB_529FEA8EE33F]

The potential for therapy of immune disorders with gene therapy

Candotti F., 2000/12. Pediatr Clin North Am, 47 (6) pp. 1389-407.

[Pmid][serval:BIB_DE0BE215637E]

Combined immunodeficiencies due to defects in signal transduction: defects of the gammac-JAK3 signaling pathway as a model

Notarangelo L. D., Giliani S., Mella P., Schumacher R. F., Mazza C., Savoldi G., Rodriguez-Perez C., Badolato R., Mazzolari E., Porta F. et al., 2000/08. Immunobiology, 202 (2) pp. 106-19.

[Pmid][serval:BIB_575EFE9170C5]

Molecular modeling of the Jak3 kinase domains and structural basis for severe combined immunodeficiency

Vihinen M., Villa A., Mella P., Schumacher R. F., Savoldi G., O'Shea J. J., Candotti F., Notarangelo L. D., 2000/08. Clin Immunol, 96 (2) pp. 108-18.

[DOI][Pmid][serval:BIB_8B83968DE812]

Hierarchy of protein tyrosine kinases in interleukin-2 (IL-2) signaling: activation of syk depends on Jak3; however, neither Syk nor Lck is required for IL-2-mediated STAT activation

Zhou Y. J., Magnuson K. S., Cheng T. P., Gadina M., Frucht D. M., Galon J., Candotti F., Geahlen R. L., Changelian P. S., O'Shea J. J., 2000/06. Mol Cell Biol, 20 (12) pp. 4371-80. Peer-reviewed.

[Pmid][serval:BIB_2BF65B5FCBFD]

Development of autologous T lymphocytes in two males with X-linked severe combined immune deficiency: molecular and cellular characterization

Mella P., Imberti L., Brugnoni D., Pirovano S., Candotti F., Mazzolari E., Bettinardi A., Fiorini M., De Mattia D., Martire B. et al., 2000/04. Clin Immunol, 95 (1 Pt 1) pp. 39-50.

[DOI][Pmid][serval:BIB_9D05480370AB]

Use of a herpes thymidine kinase/neomycin phosphotransferase chimeric gene for metabolic suicide gene transfer

Candotti F., Agbaria R., Mullen C. A., Touraine R., Balzarini J., Johns D. G., Blaese R. M., 2000/04. Cancer Gene Ther, 7 (4) pp. 574-80.

[DOI][Pmid][serval:BIB_A9DF1CFB10C7]

Complex effects of naturally occurring mutations in the JAK3 pseudokinase domain: evidence for interactions between the kinase and pseudokinase domains

Chen M., Cheng A., Candotti F., Zhou Y. J., Hymel A., Fasth A., Notarangelo L. D., O'Shea J. J., 2000/02. Mol Cell Biol, 20 (3) pp. 947-56.

[Pmid][serval:BIB_9E0A35C08515]

JAK3-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY

Notarangelo Luigi D., Candotti Fabio, 2000/02/01. Immunology and Allergy Clinics, 20 (1) pp. 97-111.

[DOI][serval:BIB_91E68540067C]

Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy

Otsu M., Anderson S. M., Bodine D. M., Puck J. M., O'Shea J. J., Candotti F., 2000/02. Mol Ther, 1 (2) pp. 145-53.

[DOI][Pmid][serval:BIB_06597D93FA4D]

Complete genomic organization of the human JAK3 gene and mutation analysis in severe combined immunodeficiency by single-strand conformation polymorphism

Schumacher R. F., Mella P., Badolato R., Fiorini M., Savoldi G., Giliani S., Villa A., Candotti F., Tampalini A., O'Shea J. J. et al., 2000/01. Hum Genet, 106 (1) pp. 73-9.

[Pmid][serval:BIB_D62095319BA7]

Efficient gene transfer to human peripheral blood monocyte-derived dendritic cells using human immunodeficiency virus type 1-based lentiviral vectors

Chinnasamy N., Chinnasamy D., Toso J. F., Lapointe R., Candotti F., Morgan R. A., Hwu P., 2000. Hum Gene Ther, 11 (13) pp. 1901-9.

[DOI][Pmid][serval:BIB_BEA371CC0949]

In vivo competitive studies between normal and common gamma chain-defective bone marrow cells: implications for gene therapy

Otsu M., Sugamura K., Candotti F., 2000. Hum Gene Ther, 11 (14) pp. 2051-6.

[DOI][Pmid][serval:BIB_A5261E569058]

Lentiviral-mediated gene transfer into human lymphocytes: role of HIV-1 accessory proteins

Chinnasamy D., Chinnasamy N., Enriquez M. J., Otsu M., Morgan R. A., Candotti F., 2000. Blood, 96 (4) pp. 1309-16.

[Pmid][serval:BIB_1E8925911DE1]

1999

Prenatal diagnosis of JAK3 deficient SCID

Schumacher R. F., Mella P., Lalatta F., Fiorini M., Giliani S., Villa A., Candotti F., Notarangelo L. D., 1999/07. Prenat Diagn, 19 (7) pp. 653-6.

[Pmid][serval:BIB_00E0D3209B4F]

Retrovirus-mediated WASP gene transfer corrects defective actin polymerization in B cell lines from Wiskott-Aldrich syndrome patients carrying 'null' mutations

Candotti F., Facchetti F., Blanzuoli L., Stewart D. M., Nelson D. L., Blaese R. M., 1999/06. Gene Ther, 6 (6) pp. 1170-4.

[DOI][Pmid][serval:BIB_AB57174DB6CC]

Autosomal SCID caused by a point mutation in the N-terminus of Jak3: mapping of the Jak3-receptor interaction domain

Cacalano N. A., Migone T. S., Bazan F., Hanson E. P., Chen M., Candotti F., O'Shea J. J., Johnston J. A., 1999. EMBO J, 18 (6) pp. 1549-58.

[DOI][Pmid][serval:BIB_779B07998F5A]

Enzyme prodrug gene therapy: synergistic use of the herpes simplex virus-cellular thymidine kinase/ganciclovir system and thymidylate synthase inhibitors for the treatment of colon cancer

Wildner O., Blaese R. M., Candotti F., 1999. Cancer Res, 59 (20) pp. 5233-8.

[Pmid][serval:BIB_E534828DBF6B]

Gene therapy

Candotti Fabio, Blaese R. Michael, 1999. dans Ochs Hans, Smith C.I. Edvard, Puck Jennifer (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

[serval:BIB_2DE1B16BEE6D]

Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials

Onodera M., Nelson D. M., Sakiyama Y., Candotti F., Blaese R. M., 1999. Acta Haematol, 101 (2) pp. 89-96.

[Pmid][serval:BIB_39E9AB4FA040]

Prenatal molecular diagnosis of Wiskott-Aldrich syndrome by direct mutation analysis

Giliani S., Fiorini M., Mella P., Candotti F., Schumacher R. F., Wengler G. S., Lalatta F., Fasth A., Badolato R., Ugazio A. G. et al., 1999/01. Prenat Diagn, 19 (1) pp. 36-40.

[Pmid][serval:BIB_662364299D70]

Retroviral-mediated transfer and expression of the common gamma chain into human hematopoietic progenitors

Candotti F., Onodera M., Knazek R. A., Blaese R. M., 1999. Acta Haematol, 101 (2) pp. 106-10.

[Pmid][serval:BIB_B4CBBF914668]

Severe combined immune deficiency due to defects of the JAK3 tyrosine kinase

Candotti Fabio, Villa Anna, Notarangelo Luigi D., 1999. pp. 111-120 dans Ochs Hans, Smith C.I. Edward, Puck Jennifer (eds.) Primary Immunodeficiency Diseases, A molecular and Genetic Approach, Oxford University Press.

[serval:BIB_42E749A4C48B]

1998

Molecular and biochemical characterization of JAK3 deficiency in a patient with severe combined immunodeficiency over 20 years after bone marrow transplantation: implications for treatment

Bozzi F., Lefranc G., Villa A., Badolato R., Schumacher R. F., Khalil G., Loiselet J., Bresciani S., O'Shea J. J., Vezzoni P. et al., 1998/09. Br J Haematol, 102 (5) pp. 1363-6.

[Pmid][serval:BIB_B250F09EE815]

Generation of a conditionally neo(r)-containing retroviral producer cell line: effects of neo(r) on retroviral titer and transgene expression

Wildner O., Candotti F., Krecko E. G., Xanthopoulos K. G., Ramsey W. J., Blaese R. M., 1998/05. Gene Ther, 5 (5) pp. 684-91.

[DOI][Pmid][serval:BIB_0AE22EF29B81]

Development of autologous, oligoclonal, poorly functioning T lymphocytes in a patient with autosomal recessive severe combined immunodeficiency caused by defects of the Jak3 tyrosine kinase

Brugnoni D., Notarangelo L. D., Sottini A., Airo P., Pennacchio M., Mazzolari E., Signorini S., Candotti F., Villa A., Mella P. et al., 1998. Blood, 91 (3) pp. 949-55.

[Pmid][serval:BIB_5E1097D64DA5]

Gene therapy of primary immunodeficiencies

Candotti F., Blaese R. M., 1998. Springer Semin Immunopathol, 19 (4) pp. 493-508.

[Pmid][serval:BIB_BA5B92FBE6D6]

Gene therapy: current status, perspectives and problematic issues

Candotti Fabio, Notarangelo Luigi D., 1998. dans Primum non nocere today. 2nd Edition, Elsevier Science B.V..

[serval:BIB_3CCFEC1C4B94]

Prenatal diagnosis in 26 fetuses at-risk for severe primary immune deficiencies

Cominotti A., Giliani S., Zucca S., Brugnoni D., Airó P., Candotti F., Fiorini M., Mella P., Badolato R., Schumacher F. et al., 1998. Ital J Pediat, 24 (1) pp. 117-121. Peer-reviewed.

[WoS][serval:BIB_1EF38C71F0F5]

Severe combined immune deficiencies due to defects of the common gamma chain-JAK3 signaling pathway

Candotti F., O'Shea J. J., Villa A., 1998. Springer Semin Immunopathol, 19 (4) pp. 401-15.

[Pmid][serval:BIB_6AC0305EFDBD]

1997

Advances in the understanding of cytokine signal transduction: the role of Jaks and STATs in immunoregulation and the pathogenesis of immunodeficiency

O'Shea J. J., Notarangelo L. D., Johnston J. A., Candotti F., 1997/11. J Clin Immunol, 17 (6) pp. 431-47.

[Pmid][serval:BIB_D8ECA633403E]

Gene therapy of ADA deficiency: the first clinical trial

Candotti Fabio, Notarangelo Luigi D., 1997. Minerva Biotec, 9 (4) pp. 210-216. Peer-reviewed.

[WoS][serval:BIB_9EB9D69A7FCD]

Interleukin-4 signaling in B lymphocytes from patients with X-linked severe combined immunodeficiency

Taylor N., Candotti F., Smith S., Oakes S. A., Jahn T., Isakov J., Puck J. M., O'Shea J. J., Weinberg K., Johnston J. A., 1997. J Biol Chem, 272 (11) pp. 7314-9.

[Pmid][serval:BIB_2894D0AF945E]

Mutation analysis of IL2RG in human X-linked severe combined immunodeficiency

Puck J. M., Pepper A. E., Henthorn P. S., Candotti F., Isakov J., Whitwam T., Conley M. E., Fischer R. E., Rosenblatt H. M., Small T. N. et al., 1997. Blood, 89 (6) pp. 1968-77.

[Pmid][serval:BIB_11F10D33E630]

Retroviral transfer of acid alpha-glucosidase cDNA to enzyme-deficient myoblasts results in phenotypic spread of the genotypic correction by both secretion and fusion

Zaretsky J. Z., Candotti F., Boerkoel C., Adams E. M., Yewdell J. W., Blaese R. M., Plotz P. H., 1997. Hum Gene Ther, 8 (13) pp. 1555-63.

[DOI][Pmid][serval:BIB_BC0C5E15E02C]

Structural and functional basis for JAK3-deficient severe combined immunodeficiency

Candotti F., Oakes S. A., Johnston J. A., Giliani S., Schumacher R. F., Mella P., Fiorini M., Ugazio A. G., Badolato R., Notarangelo L. D. et al., 1997. Blood, 90 (10) pp. 3996-4003.

[Pmid][serval:BIB_303027D0E49C]

1996

Signaling via IL-2 and IL-4 in JAK3-deficient severe combined immunodeficiency lymphocytes: JAK3-dependent and independent pathways

Oakes S. A., Candotti F., Johnston J. A., Chen Y. Q., Ryan J. J., Taylor N., Liu X., Hennighausen L., Notarangelo L. D., Paul W. E. et al., 1996/12. Immunity, 5 (6) pp. 605-15.

[Pmid][serval:BIB_A8ECD31EBE04]

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